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Assisgment by panchalpk38 is a document available to read on EtoBox.
Duchenne Muscular Dystrophy (DMD) is a severe genetic disorder caused by mutations in the dystrophin gene, leading to muscle degeneration and premature death. Gene therapy approaches, such as AAV-mediated micro dystrophin therapy and exon skipping, aim to restore dystrophin expression or compensate for its loss, offering potential disease-modifying treatments. Despite challenges like immune responses and delivery efficiency, advancements in gene therapy may transform DMD into a manageable condition.
- Author
- panchalpk38
- Language
- EN